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BMN165 /day

Phase 3

Phenylketonuria (PKU) | Small molecule | Rare Disease |BioMarin Pharmaceutical Inc.|Last Updated: May 21, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment215
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUPHASE3 COMPLETED 215 — — Jul 29, 2013Feb 5, 2019May 21, 202129 United States
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Study Endpoints
Primary Endpoints
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2
At Part 2 baseline and change from baseline at Part 2 week 8

Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo.

Secondary Endpoints
Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2
At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4
At Part 4 week 57, 105, 161, 209 and 249
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2
At Part 2 baseline and change from baseline at Part 2 week 8
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BMN165 20mg/dayACTIVE_COMPARATORBMN165 20mg/day self-administered daily
20 mg/day PlaceboPLACEBO_COMPARATOR20 mg/day Placebo self-administered daily
BMN165 40mg/dayACTIVE_COMPARATORBMN165 40mg/day self-administered daily
40 mg/day PlaceboPLACEBO_COMPARATOR40 mg/day Placebo self-administered daily
Interventions
NameTypeDescription
BMN165 20mg/dayDRUGBMN165 20mg/day self-administered daily
BMN165 40mg/dayDRUGBMN165 40mg/day self-administered daily
PlaceboDRUGNon-drug placebo, self-administered daily
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites29

INCLUSION CRITERIA Individuals eligible to participate in this study must meet all of the following criteria: * Have completed a prior BMN 165 study (PAL-003, 165-205, or 165-301) prior to screening * Have had a stable BMN 165 dose regimen for at least 14 days prior to screening * Are at least 18 ...

Countries:United States
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Competitive Landscape -Phenylketonuria 12 trials
CompanyTickerTrialsLead PhaseDrugs
BioMarin Pharmaceutical Inc.BMRN5PHASE3Pegvaliase, BMN 307
PTC Therapeutics, Inc.PTCT2PHASE3PTC923, Sepiapterin
Sanofi SA Sponsored ADRSNY1PHASE1SAR444836
Agios Pharmaceuticals, Inc.AGIO1PHASE1AG-181
Illumina, Inc.ILMN1 — Undisclosed
Q32 Bio IncQTTB1 — HMI-102
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